Race, Funding, and Access to High Quality Care

The agonizing pain of sickle cell disease

Sitting across the room from a young, typically articulate patient who is in agony is a common and difficult moment in our sickle cell clinic. While the pain is unbearable for the patients, sitting in that clinic is an uncomfortable experience for providers on many levels. Years ago, before we had a dedicated clinic to provide rapid care, seeing a suffering patient in clinic meant sending the patient to the emergency department knowing they may wait for several hours for a bed before receiving treatment that would relieve their pain—pain that is well known to be worse than renal colic and labor pains.

40 years later, still masking the pain

With cooperation from our ED colleagues and the availability of the sickle infusion clinic we can provide patients with treatment quickly, but the frustration remains. The therapy we currently provide for patients who are having a painful crisis, the most common complication of sickle cell disease, is the same therapy that we’ve been providing for the last 40 years.

Despite being the first molecular disease described by Linus Pauling in 1949, and despite having an excellent understanding of the pathophysiology of sickling, we have no available treatments to stop a painful crisis. We simply mask the pain with strong opioids that cause side effects and wait it out along with our patients who often suffer for days.

There are only two FDA approved medications for the management of sickle cell disease (SCD), one approved in 1998, hydroxyurea, and the second approved in 2017, L-glutamine. Both medications have been shown to prevent painful episodes. Hydroxyurea, with which we have years of experience, is a great therapy and decreases painful episodes by 50% and prolongs overall survival. L-glutamine, an amino acid whose mechanism of benefit in sickle cell disease is unclear, has been shown to decrease painful episodes by 25% when compared to placebo. Stem cell transplant is a potential curative therapy but is only paid for by CMS if done as part of a research protocol.

Recent data shows that dramatic gains in survival for children that started in the 1980’s are restricted to children. Despite the available therapies, overall mortality rates for adults remain unchanged, and overall survival by some reports may be decreasing.

Race plays a role

Why hasn’t there been more progress in the study of treatments for people with sickle cell disease?  Some propose that race plays a role as noted in this recent 1A NPR broadcast. Sickle cell disease predominantly effects minority populations in the US, especially those of African descent.

 In 2006, Smith et al published a paper comparing Federal and Foundation funding for sickle cell disease and cystic fibrosis research. The disparity was striking with federal funding of $4267/person for cystic fibrosis compared to $1125/person for sickle cell disease. In 2003 the Cystic Fibrosis Foundation raised $152 million and the Sickle Cell Disease Association of America raised just under $500,000.

The two diseases affect different populations, but with race as one of the main differences, it’s very hard to ignore it as a factor in these numbers. This health inequity persists— there is no question that more research dollars are necessary to discover and study new therapies for SCD.

Lack of access to high quality care

Another disparity exists which effects patients around the US every day and puts lives at risk. The lack of access to high quality care leads to early mortality and needless suffering. Sickle cell disease causes significant morbidity and mortality, some of which is not preventable with our current technology, but there are many patients who live in areas without access to the best technology and knowledgeable providers. Whether it is the death of a young mother in Kansas, a child who dies before their 18^th birthday, or the death of a rap artist, many of these deaths are preventable.

There are over 100 comprehensive treatment centers in the cystic fibrosis network for the approximately 35,000 people with the disease in the US. There is no network of comprehensive centers for the 100,000 people living with SCD in the US.

The expectation is that primary care providers know how to treat this disease, yet there is data showing that most primary care providers are uncomfortable providing even the most routine care to this patient population. With lack of access to high quality and knowledgeable providers, it is no wonder that hydroxyurea is underutilized in this patient population.

We anticipate that new therapies, including gene therapy, will be available to people living with SCD in the coming years, which have the potential to dramatically improve quality of life.

However, without changes in funding and a systematic change in health care delivery to this underserved population those therapies may never reach the most vulnerable of patients.